Adeno-associated viral compositions for IDS gene transfer and methods of use thereof

Adeno-associated viral compositions for IDS gene transfer and methods of use thereof

Adeno-associated virus (AAV) compositions that can restore IDS gene function of a cell and methods for using these AAV compositions to treat conditions associated with reduced IDS gene function (e.g., Hunter Syndrome) are provided. Compositions, systems, and methods for making the AAV compositions a...

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Bibliographische Detailangaben
Hauptverfasser: SMITH LAURA JANE, GINGRAS JACQUES, BURNHAM BRIAN, PATEL KETAN, WHITE YANNICK, DOLIVE STEPHEN N, VAN LIESHOUT, LEON
Format: Patent
Sprache:chi ; eng
Schlagworte:
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BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
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Zusammenfassung:Adeno-associated virus (AAV) compositions that can restore IDS gene function of a cell and methods for using these AAV compositions to treat conditions associated with reduced IDS gene function (e.g., Hunter Syndrome) are provided. Compositions, systems, and methods for making the AAV compositions are also provided. 提供了可以恢复细胞的IDS基因功能的腺相关病毒(AAV)组合物以及用于使用这些AAV组合物治疗与IDS基因功能的降低相关的病症(例如，亨特综合征)的方法。还提供了用于制备所述AAV组合物的组合物、系统和方法。